13 hours ago Fruit fly larva are used to study stem cells key features. Credit: Wikipedia

The study, performed with fruit flies, describes a gene that determines whether a specialized cell conserves the capacity to become a stem cell again. Unveiling the genetic traits that favour the retention of stem cell properties is crucial for regenerative medicine. Published in Cell Reports, the article is the fruit of collaboration between researchers at IRB Barcelona and CSIC.

One kind of stem cell, those referred to as 'facultative', form parttogether with other cellsof tissues and organs. There is apparently nothing that differentiates these cells from the others. However, they have a very special characteristic, namely they retain the capacity to become stem cells again. This phenomenon is something that happens in the liver, an organ that hosts cells that stimulate tissue growth, thus allowing the regeneration of the organ in the case of a transplant. Knowledge of the underlying mechanism that allows these cells to retain this capacity is a key issue in regenerative medicine.

Headed by Jordi Casanova, research professor at the Instituto de Biologa Molecular de Barcelona (IBMB) of the CSIC and at IRB Barcelona, and by Xavier Franch-Marro, CSIC tenured scientist at the Instituto de Biologa Evolutiva (CSIC-UPF), a study published in the journal Cell Reports reveals a mechanism that could explain this capacity. Working with larval tracheal cells of Drosophila melanogaster, these authors report that the key feature of these cells is that they have not entered the endocycle, a modified cell cycle through which a cell reproduces its genome several times without dividing.

"The function of endocycle in living organisms is not fully understood," comments Xavier Franch-Marro. "One of the theories is that endoreplication contributes to enlarge the cell and confers the production of high amounts of protein". This is the case of almost all larval cells of Drosophila.

The scientists have observed that the cells that enter the endocycle lose the capacity to reactivate as stem cells. "The endocycle is linked to an irreversible change of gene expression in the cell," explains Jordi Casanova, "We have seen that inhibition of endocycle entry confers the cells the capacity to reactivate as stem cells".

Cell entry into the endocycle is associated with the expression of the Fzr gene. The researchers have found that inhibition of this gene prevents this entry, which in turn leads to the conversion of the cell into an adult progenitor that retains the capacity to reactivate as a stem cell. Therefore, this gene acts as a switch that determines whether a cell will enter mitosis (the normal division of a cell) or the endocycle, the latter triggering a totally different genetic program with a distinct outcome regarding the capacity of a cell to reactivate as a stem cell.

Explore further: Autophagy helps fast track stem cell activation

More information: Specification of Differentiated Adult Progenitors via Inhibition of Endocycle Entry in the Drosophila Trachea, Nareg J.-V. Djabrayan, Josefa Cruz, Cristina de Miguel, Xavier Franch-Marro, Jordi Casanova, Cell Reports (2014) DOI: dx.doi.org/10.1016/j.celrep.2014.09.043

In spite of considerable research efforts around the world, we still do not know the determining factors that confer stem cells their main particular features: capacity to self-renew and to divide and proliferate. The scientist ...

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A mechanism that allows a differentiated cell to reactivate as a stem cell revealed

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Researchers at Washington University have figured out how to scratch off a tiny piece of flesh from a persons forearm, isolate the skin cells and turn those cells into neurons the brain cells that transmit information in the form of electrical signals throughout the body.

The research is significant because it could put researchers on a path to treating certain diseases of the brain, including Alzheimers, ALS, Parkinsons and others.

Washington Universitys research, published Oct. 22 in the journal Neuron, relates specifically to Huntingtons disease, an inherited illness that affects an estimated 30,000 people in the U.S.

Huntingtons is caused when neurons in the brain start to malfunction and die. Initially, sufferers often experience problems with coordination and learning new information. As more neurons die, symptoms worsen and can be fatal.

Andrew Yoo, an assistant professor of developmental biology, explained that skin cells have the same DNA as brain cells.

To get skin cells to begin acting like brain cells, Yoos team at the Washington University medical school was able to create a process in which skin cells were reprogrammed into brain cells.

Basically we were able to pull a genetic trick to generate neurons, Yoo said.

The reprogrammed cells are a specific type that play a large role in controlling movement. They are typically the cells affected by Huntingtons disease.

The next step for researchers, Yoo said, is to re-create the conditions for Huntingtons in a Petri dish to study healthy brain cells alongside malfunctioning ones to get a better understanding of what causes the disease.

From there, Yoo said its possible that researchers develop a way to treat the illness.

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Move over stem cells. A different kind of cellular alchemy is allowing cells to be converted directly into other tissues to treat disease or mend injuries.

Stem cells have long been touted as the future of regenerative medicine as they can multiply indefinitely and be turned into many different cell types. Ideally, this would take a personal approach a patient's own cells would be converted into whatever type of cell is required to fix their injury or treat their symptoms. Earlier this year, for instance, people with age-related macular degeneration, the most common cause of blindness in the West, had retinal cells made from their own stem cells injected into their eyes.

Mature cells can be converted into stem cells by exposing them to a cocktail of chemicals that reverts them back to an embryonic-like state. Another set of chemicals is then used to turn the cells into the desired tissue type.

Skipping the stem cell stage would be more efficient, says Andrew Yoo of Washington University in St Louis, Missouri, and would reduce the chance that the new tissue could grow into a tumour a risk with stem cells because of their capacity to regenerate.

Yoo has now managed to do just that, using a process known as "transdifferentiation". His team have turned human skin cells into medium spiny neurons, the cells that go wrong in Huntington's disease.

To the skin cells, the team added two short snippets of genetic material called microRNAs. MicroRNAs are signalling molecules and the two they picked turn on genes in brain cells during embryonic development. They also added four transcription factors another kind of signalling molecule to turn on genes normally active in medium spiny neurons.

Within four weeks the skin cells had changed into MSNs. When put into the brains of mice, the cells survived for at least six months and made connections with the native tissue. "This is a very cool result," says Ronald McKay of the Lieber Institute for Brain Development in Baltimore.

The team's next step is to transplant the cells into mice with a version of Huntington's to see if the new neurons reduce their symptoms.

"Being able to produce cells with medium spiny neuron characteristics directly without first having to generate stem cells is impressive," says Edward Wild of University College London. "Using this offers the tantalising prospect of cell replacement treatments."

Wild points out, however, that before this approach can be used on people with Huntington's, researchers would first have to correct the faulty genetic mutation in their skin cells. And while medium spiny neurons are the first to degenerate in the disease, other brain cells may also be affected. "When it comes to cell replacement we should probably be aiming for a cocktail of cells," says Wild.

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Scientists have grown miniature stomachs in a lab dish using stem cells, and are already using them to study stomach cancer. They hope they can grow patches to fix ulcers, find new drugs to treat and even prevent stomach cancer, and perhaps even grow replacement stomachs some day.

They discovered that the bacteria that cause stomach cancer begin doing their dirty work almost immediately, attaching to the stomach lining and causing tumors to start growing in response. Helicobacter pylori causes many, if not most, cases of stomach cancer, which affects more than 22,000 Americans a year and kills half of them. Stomach cancer is a major killer globally, affecting close to a million people a year and killing more than 70 percent of them.

And the team grew their mini-stomachs using two different types of stem cells human embryonic stem cells, grown from very early human embryos, but also induced pluripotent stem cells or iPS cells, which are made by tricking bits of skin or other tissue into acting like a stem cell.

In our hands they worked exactly the same, James Wells of Cincinnati Childrens Hospital Medical Center, who led the research. Both were able to generate, in a petri dish, human stomach tissue.

Immunofluorescent image of human stomach tissue made using stem cells

Stem cells are the body's master cells. Embryonic stem cells and iPS cells are both pluripotent meaning they can give rise to any tissue in the body. They've been used to grow miniature human livers, retinas, brain tissue and have been injected into eyes to treat eye disease.

Growing anything close to a real stomach or even a patch for an ulcer is a long way off. The gastric organoids Wellss team made the name up are just about the size of a BB bullet.

Its not easy getting stem cells to do what you want them to do. Wells and his team, including graduate student Kyle McCracken, had to use various growth factors and chemicals, each introduced at precisely the right time, to coax the cells into becoming three-dimensional blobs of stomach tissue. The stomach is a complex organ, with layers of muscle cells, cells that make up the stomach lining and glands that secrete proteins and acid to digest food.

"The bacteria immediately know what to do and they behaved as if they were in the stomach.

But the process worked, and the mini-stomachs look just like stomach tissue, the team reports in this weeks issue of the journal Nature.

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Mini-Stomachs Let Scientists Study Ulcers in a Lab Dish

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Copy of PhytoScience Philippines Celeb Share good effect of Stem Cell Therapy
PHYTOSCIENCE DOUBLE STEM CELL removes the apperance of age lines and restore smoth, radiant, youthful looking skin! LOOK YOUNGER REDUCE THE LOOK OF WRINKLES LINES ...

By: Emmanuel Villamor Jr

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The U.S. Food and Drug Administration today announced it has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics.

The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products.

The FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program, said Gayatri R. Rao, M.D., director of the FDAs Office of Orphan Product Development. The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.

The program is administered through the FDAs Orphan Products Grants Program. This program was created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the program has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare diseases and has been used to bring more than 50 products to marketing approval.

A panel of independent experts with experience in the disease-related fields reviewed the grant applications and made recommendations to the FDA.

The 2014 grant recipients are:

For the grants program therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the United States. There are about 7,000 rare diseases and conditions, according to the National Institutes of Health. In total, nearly 30 million Americans suffer from at least one rare disease.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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New York, New York (PRWEB) October 29, 2014

Stem cell therapy is the future of foot pain treatment. New York podiatrists at Adler Footcare are using ethical stem cell treatments for foot problems to help speed healing, minimize pain, and reduce swelling.

Stem cells are cells that havent quite yet determined their role in the body. This gives them the ability to turn into anything. The treatment is being used for problems causing foot pain, such as Achilles tendonitis, plantar fasciitis, and arthritis of the first toe joint. Stem cells help regenerate new cartilage and helps tissue heal much quicker.

"Stem cells turn into everything," said Dr. Jeffrey Adler, Medical/Surgical Director & Owner of Adler Footcare. "So basically, if the damage is due to cartilage, they turn into cartilage. If the damage is due to soft tissue, they turn into soft tissue. Its the Swiss army knife of treatments."

The stem cells are not live embryos, but instead are generated from the placenta and ethically obtained during the C-sections of live births. The women who the cells are taken from are screened and tested for any communicable diseases beforehand.

Stem cell therapy uses a minimally invasive technique to inject the cells directly into the area where the patient is feeling the foot pain. Fluoroscopy is used to determine the exact position for injection. When stem cell therapy is used healing occurs twice as fast. As the tissues are regenerated and the swelling is minimized, the patient is able to experience more range of motion, less post-operative pain, and less inflammation.

The New York podiatrists at Adler Footcare have been using stem cell therapy for 2 years. They continue to stay up-to-date on the process and have seen only positive results.

To learn more about stem cell treatment for foot pain, contact a New York podiatrist at Adler Footcare.

About Dr. Jeffrey L. Adler

Dr. Jeffrey L. Adler, Medical/Surgical Director and Owner of Adler Footcare of Greater New York has been practicing podiatric medicine since 1979 and has performed thousands of foot and ankle surgeries. Dr. Adler is board certified in Podiatric Surgery and Primary Podiatric Medicine by the American Board of Multiple Specialties in Podiatry. Dr. Adler is also a Professor of Minimally Invasive Foot Surgery for the Academy of Ambulatory Foot and Ankle Surgeons. As one of only several in the country who perform minimally invasive podiatric surgery, Dr. Adlers patients enjoy significantly reduced recovery times.

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The Miracle of Stem Cell Therapy at Adler Footcare Regenerates Cells, Heals Foot Pain

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OTTAWA - A coalition of Canadian stem cell advocates, researchers and charities is calling for $1.5 billion in private and public funding for stem cell therapy over the next 10 years.

The coalition's action plan is aimed at cementing Canada's reputation as a stem cell leader, one that uses stem cell science to reduce suffering and death from cardiovascular diseases, cancer, diabetes, vision loss, spinal cord injuries and other conditions.

James Price, the president and CEO of the Canadian Stem Cell Foundation, says the action plan could help millions of people with new, life-changing therapies.

The action plan's call for funding includes a $50 million scaled annual average commitment by the federal government.

The Centre for Commercialization of Regenerative Medicine estimates the action plan could also create more than 12,000 jobs due to the growth of existing companies and the development of new enterprises aimed at global markets.

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A gene previously linked to risk-taking and aggression appears to contribute to extreme violent behavior as well, according to a study that may open the way for treatments to prevent such behavior.

The gene, identified in an analysis of more than 700 criminal offenders, is one of two spotlighted in a study published today in the journal Molecular Psychiatry. The second gene is associated with attention deficit hyperactivity disorder, the authors wrote.

While researchers have attributed about 50 percent of severe antisocial or criminal behavior to genetics rather then environmental factors, theyve identified only a few genes that may be involved. While the latest research wont help predict which individuals might commit violence, it may lead to potential treatments, the researchers said.

So far, nobody had found any gene associated with severe violent crime, says Jari Tiihonen, a psychiatrist at Karolinska Institute in Stockholm, who led the study. We wanted to try to find it, and we found two.

Tiihonen and colleagues analyzed the genomes of 895 Finnish prisoners across the 19 largest prisons in Finland. The participants included nonviolent offenders involved in cases such as drunk-driving and property theft, those convicted of violent crimes including murder, and extremely violent offenders, those responsible for at least 10 violent crimes.

The researchers compared the prisoner results with a control population of Finnish citizens. By doing so, they identified two gene variants present in those with extreme violent behavior, but not in those without.

The first is a mutated variant of monoamine oxidase A, or MAOA, which breaks down chemicals, such as dopamine and serotonin, that communicate information among brain cells. In 1993, MAOA was linked to impulsive aggression in five males from a single family and dubbed the warrior gene by the media.

The warrior gene finding has already begun to influence U.S. courts, yet there are strongly conflicting opinions about how much of a role the genetic basis of crime should play in the courtroom. It is too soon to know if this finding might also make its way into the justice system, but Tiihonen hopes it will not.

No single risk factor, whether a gene variant or brain damage from a car accident or whatever, should affect a sentence, Tiihonen said, adding that a persons total mental capacity should be judged in assessing a sentence.

J.C. Barnes, a criminologist at the University of Cincinnati who wasnt involved in the study, agrees. Interactions between genes and the environment make it impossible to say this person will develop (violent behavior), just based on knowing a handful of genes, or even thousands of genes, he says.

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Extreme Violent Crimes Tied to Gene in Study of Criminals

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29-Oct-2014

Contact: Anastasia Greenebaum agreenebaum@simonsfoundation.org 212-524-6097 Simons Foundation

A genetic autism study of unprecedented scope and power has uncovered more than two-dozen high-confidence risk genes for the disorder. It offers compelling evidence that spontaneous, or de novo, mutations contribute to autism in at least 27 percent of families in which the parents and siblings are unaffected.

The new research has also established conclusively that 'higher-IQ' autism, which mostly affects boys, has a different genetic basis from 'lower-IQ' autism, which commonly affects both boys and girls.

The researchers, whose findings will be published by Nature on 29 October, 2014, at http://dx.doi.org/10.1038/nature13908, sequenced the whole exomes the protein-coding regions of the genome of 2,515 families from the Simons Simplex Collection, a large repository of genetic, biological and phenotypic data from 'simplex' families, which consist of one child with autism, unaffected parents and usually at least one unaffected sibling.

The study, carried out at three different universities across the country, pinpointed seven genes that have mutations in three or more children with autism, implicating these genes in the disorder with near certainty. It also identified another 20 genes with mutations in two children. Each of these genes has more than a 90 percent chance of being a true autism gene, the researchers reported in their paper, 'The contribution of de novo coding mutations to autism spectrum disorder.'

"We have a set of genes for which now, if people see a likely gene-disrupting mutation when sequencing a young child, there's a high risk of the child developing autism, and that, to my mind, is pretty powerful stuff," says Evan Eichler of the University of Washington, a Simons Foundation Autism Research Initiative (SFARI) Investigator who leads one of the laboratories that contributed to the study. "Recognizing this early on may allow for earlier interventions, such as behavioral therapies, improving outcomes in children."

The exome analysis was also carried out in the laboratories of SFARI Investigators Michael Wigler of Cold Spring Harbor Laboratory in New York and Matthew State of the University of California, San Francisco.

Although the researchers at the three different universities might be assumed to be competitors, in an unusual move they have published the results of their exome-sequencing studies in a single joint paper. "That seemed to us to make the most sense scientifically, though not from the standpoint of getting credit," Eichler says. "We felt that it would create the biggest benefit for the community of autism researchers."

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What is Synthetic Biology?: Engineering Life and Livelihoods
It has been referred to as extreme genetic engineering and the new frontier of biotechnology. What is "SynBio", and how will it affect the food we eat and the farmers who provide it? This short...

By: ETC Group

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Genetic Engineering and Plastic Surgery in India
Prime Minister Modi #39;s literal interpretation of Hindu Scriptures.

By: Muneeb Haroon

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29-Oct-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News @LiebertOnline

New Rochelle, October 29, 2014 Cities around the world are increasingly making urban data freely available to the public. But is the content or structure of these vast data sets easy to access and of value? A new study of more than 9,000 data sets from 20 cities presents encouraging results on the quality and volume of the available data and describes the challenges and benefits of analyzing and integrating these expanding data sets, as described in an article in Big Data, the highly innovative, peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The Open Access article is available free on the Big Data website.

In the article "Structured Open Urban Data: Understanding the Landscape," Luciano Barbosa and Marcos Vieira, IBM Research, Brazil, and Kien Pham, Claudio Silva, and Juliana Freire, New York University School of Engineering and NYU Center for Urban Science and Progress, NY, present several promising findings. These include a steadily increasing volume of open urban data, the ability to integrate different data sets, and the finding that much of the available data is published in standard types of formats. The authors also discuss the main challenges that make it difficult to take full advantage of these data sources.

"Big urban data is a powerful new phenomenon that has the potential to transform everyday lives of hundreds of millions of people quickly via personal devices that integrate, filter, and create useful personalized information. This paper documents the sources and value of these data," says Big Data Editor-in-Chief Vasant Dhar, Co-Director, Center for Business Analytics, Stern School of Business, New York University.

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About the Journal

Big Data, published quarterly in print and online, facilitates and supports the efforts of researchers, analysts, statisticians, business leaders, and policymakers to improve operations, profitability, and communications within their organizations. Spanning a broad array of disciplines focusing on novel big data technologies, policies, and innovations, the Journal brings together the community to address the challenges and discover new breakthroughs and trends living within this information. Complete tables of content and a sample issue may be viewed on the Big Data website.

About the Publisher

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29-Oct-2014

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center @sumedicine

The ability to pop a working copy of a faulty gene into a patient's genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine have devised a new way to carry out this genetic sleight of hand.

The approach differs from that of other hailed techniques because it doesn't require the co-delivery of an enzyme called an endonuclease to clip the recipient's DNA at specific locations. It also doesn't rely on the co-insertion of genetic "on" switches called promoters to activate the new gene's expression.

These differences may make the new approach both safer and longer-lasting. Using the technique, the Stanford researchers were able to cure mice with hemophilia by inserting a gene for a clotting factor missing in the animals.

"It appears that we may be able to achieve lifelong expression of the inserted gene, which is particularly important when treating genetic diseases like hemophilia and severe combined immunodeficiency," said Mark Kay, MD, PhD, professor of pediatrics and of genetics. "We're able to do this without using promoters or nucleases, which significantly reduces the chances of cancers that can result if the new gene inserts itself at random places in the genome."

Using the technique, Kay and his colleagues were able to insert a working copy of a missing blood-clotting factor into the DNA of mice with hemophilia. Although the insertion was accomplished in only about 1 percent of liver cells, those cells made enough of the missing clotting factor to ameliorate the disorder.

Kay is the senior author of the research, which will be published Oct. 29 in Nature. The lead author is postdoctoral scholar Adi Barzel, PhD.

A possible alternative to CRISPR

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New way of genome editing cures hemophilia in mice; may be safer than older method

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29-Oct-2014

Contact: Pete Farley peter.farley@ucsf.edu 415-502-6397 University of California - San Francisco @ucsf

Scores of autoimmune diseases afflicting one in 12 Americans ranging from type 1 diabetes, to multiple sclerosis (MS), to rheumatoid arthritis, to asthma mysteriously cause the immune system to harm tissues within our own bodies. Now, a new study pinpoints the complex genetic origins for many of these diseases, a discovery that may lead to better diagnosis and ultimately to improved treatments.

A team of scientists from UC San Francisco, the Broad Institute of MIT and Harvard, and Yale School of Medicine developed a new mathematical tool to more deeply probe existing DNA databases. In so doing they discovered how certain DNA variations, when inherited, are likely to contribute to disease.

By applying their method to analyzing data from previous studies of 21 different autoimmune diseases, the research team has deepened scientific understanding of the genetic underpinnings of a wide range of these disorders. They also found the specific immune cells most responsible for the diseases. Their study is published online on October 29, 2014 in Nature.

The researchers examined a wealth of data from 39 large-scale studies called genome-wide association studies (GWAS). Teams of scientists in recent years have conducted GWAS typically enlisting thousands of study participants to identify large blocks of DNA within the human genome within which genetic variants are implicated as risk factors for common diseases. But examination of GWAS data to date has seldom pointed to altered proteins, as surprisingly few protein-encoding gene variants within these broad swaths of DNA have been associated with the diseases under investigation.

Instead, the genetic risks identified through GWAS more often appear to be associated with DNA variations that do not reside within genes. The nature of this risk has defied understanding until now, fueling a perception that few medical benefits have thus far emerged from large-scale studies of human genetic variation being conducted in the wake of the initial Human Genome Project.

In the new study the researchers found that the presence of specific genetic variants in different autoimmune diseases can alter patterns of activity of genes in particular ways that affect functions of the immune system. This was true despite the fact that the genetic variants are not within genes.

To make their discoveries, the researchers developed software and used next-generation sequencing techniques to probe "epigenetic" characteristics of specialized immune cells, in which gene activity is affected without changes to the DNA sequence itself within the affected genes.

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Newswise Scores of autoimmune diseases afflicting one in 12 Americans ranging from type 1 diabetes, to multiple sclerosis (MS), to rheumatoid arthritis, to asthma mysteriously cause the immune system to harm tissues within our own bodies. Now, a new study pinpoints the complex genetic origins for many of these diseases, a discovery that may lead to better diagnosis and ultimately to improved treatments.

A team of scientists from UC San Francisco, the Broad Institute of MIT and Harvard, and Yale School of Medicine developed a new mathematical tool to more deeply probe existing DNA databases. In so doing they discovered how certain DNA variations, when inherited, are likely to contribute to disease.

By applying their method to analyzing data from previous studies of 21 different autoimmune diseases, the research team has deepened scientific understanding of the genetic underpinnings of a wide range of these disorders. They also found the specific immune cells most responsible for the diseases. Their study is published online on October 29, 2014 in Nature.

The researchers examined a wealth of data from 39 large-scale studies called genome-wide association studies (GWAS). Teams of scientists in recent years have conducted GWAS typically enlisting thousands of study participants to identify large blocks of DNA within the human genome within which genetic variants are implicated as risk factors for common diseases. But examination of GWAS data to date has seldom pointed to altered proteins, as surprisingly few protein-encoding gene variants within these broad swaths of DNA have been associated with the diseases under investigation.

Instead, the genetic risks identified through GWAS more often appear to be associated with DNA variations that do not reside within genes. The nature of this risk has defied understanding until now, fueling a perception that few medical benefits have thus far emerged from large-scale studies of human genetic variation being conducted in the wake of the initial Human Genome Project.

In the new study the researchers found that the presence of specific genetic variants in different autoimmune diseases can alter patterns of activity of genes in particular ways that affect functions of the immune system. This was true despite the fact that the genetic variants are not within genes.

To make their discoveries, the researchers developed software and used next-generation sequencing techniques to probe epigenetic characteristics of specialized immune cells, in which gene activity is affected without changes to the DNA sequence itself within the affected genes.

The team discovered that a majority of key DNA changes associated with autoimmune diseases occur in functional bits of DNA known as enhancers.

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In Autoimmune Diseases Affecting Millions, Researchers Pinpoint Genetic Risks, Cellular Culprits

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NEW Sims 3 Challenge - Perfect Genetics - C-A-S Option 1
I will be starting this challenge next month (november but no set date yet) but i want your help in choosing the founder. Over the next 3 weeks i will give y...

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CLEVELAND and COLUMBUS, Ohio >> Data published today in the journal Molecular Therapy demonstrates initial clinical proof of principle of a gene therapy to increase muscle strength and function in Becker muscular dystrophy patients. The paper, titled "A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy" describes results of the dose ascending trial to assess safety and efficacy in six patients with Becker muscular dystrophy.

All six of the patients were followed at least six months and three of the patients were followed for one year with no drug-related adverse events reported. On average, the patients showed statistically significant improvement in six minute walk distance and in stair climbing tests. Additionally, muscle biopsy histology demonstrated reductions in muscle fibrosis and central nucleation, and improved muscle fiber regeneration. Muscle satellite cells were not reduced.

Jerry Mendell, M.D., Professor at Nationwide Children's Hospital and lead author on the trial and manuscript stated, "This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease." Brian Kaspar, Ph.D., Associate Professor at Nationwide Children's Hospital, author and compensated advisor and scientific founder of Milo said, "Upon evaluating the top line data in this trial, we are particularly impressed with the effects of our gene therapeutic to express follistatin long term, not only for the functional improvement, but also in improving the state of muscle health in the patients analyzed."

The therapy, developed at Nationwide Children's Hospital by Dr. Mendell and Dr. Brian Kaspar, is based on adeno-associated virus delivery of follistatin 344 to increase muscle strength and prevent muscle wasting. It could have broad ranging applications in muscular dystrophies, cancer treatment induced muscle wasting and age related muscle wasting. The clinical study, conducted at Nationwide Children's Hospital and funded by the foundation Parent Project MD, is also evaluating safety and efficacy in nine patients with inclusion body myositis. Results of the trial in inclusion body myositis will be published in mid 2015.

The underlying intellectual property was exclusively licensed in 2012 from Nationwide Children's Hospital to Ohio-based start-up Milo Biotechnology. Milo Biotechnology has received FDA orphan designation for Becker muscular dystrophy and for Duchenne muscular dystrophy.

Milo Biotechnology CEO Al Hawkins stated "This significant and long lasting clinical effect provides the basis for planning pivotal clinical studies beginning in 2015; we want to speed delivery of this important therapy to patients with debilitating muscular dystrophies."

About Milo Biotechnology Milo Biotechnology is a clinical stage startup company developing therapies to strengthen muscle and improve the lives of patients with neuromuscular diseases. Its lead program, AAV1-FS344, leads to the local expression of follistatin, a potent myostatin inhibitor. Milo's core technology was developed at and is exclusively licensed from Nationwide Children's Hospital in Columbus, Ohio; the company is based in Cleveland, Ohio, and has received funding from JumpStart Inc., the Cuyahoga County North Coast Opportunities Fund and from NIAMS Rare Disease SBIR program. Visit http://www.milobiotechnology.com for more information

CONTACT: infomilobiotechnology.com

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Gene Therapy Increases Function in Becker Muscular Dystrophy Patients

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MITTEILUNG UEBERMITTELT VON BUSINESS WIRE. FUER DEN INHALT IST ALLEIN DAS BERICHTENDE UNTERNEHMEN VERANTWORTLICH.

Distinguished Scientist To Lead Companys Gene Therapy Research Efforts

CAMBRIDGE, Mass. --(BUSINESS WIRE)-- 23.09.2014 --

Biogen Idec (NASDAQ:BIIB) today announced that Olivier Danos, Ph.D., has joined the company as senior vice president, Gene Therapy. Dr. Danos reports directly to Douglas Williams, Ph.D., executive vice president, Research and Development, and is a member of Biogen Idecs Senior Research and Development Leadership Team.

Dr. Danos will lead the companys gene therapy research group, a team dedicated to identifying and developing new technologies for gene transfer and genome engineering. As part of his role, Dr. Danos will also identify potential collaborations that complement the companys internal capabilities and support the development of treatments across Biogen Idecs therapeutic focus areas.

Olivier is a true pioneer in the field. We believe that his expertise will help accelerate our development of a world-class gene therapy platform that combines our growing internal capabilities with collaborations with leading research organizations across the globe, said Dr. Williams. Our commitment to this important area will complement our existing capabilities in antisense, small molecule and biologics research, and enhance our ability to effectively and efficiently develop new treatments for people with serious diseases.

I have dedicated my career to advancing the use of gene therapy as a technology to develop life-saving therapies for patients, said Dr. Danos. The team at Biogen Idec shares this passion. It is their commitment to innovative science and willingness to tackle difficult medical challenges that attracted me to the company. I am excited about the opportunities before us.

Dr. Danos joins Biogen Idec from Kadmon Pharmaceuticals, where he served as senior vice president, Molecular Medicine, Synthetic Biology and Gene Regulation since 2011. In this role, he was instrumental in assembling a gene therapy program and a technology platform for the development ofcontrollablegene expression systems. Prior to Kadmon, Dr. Danos acted as the director of the Gene Therapy Consortium of the University College of London and led a gene therapy research team at the Necker Hospital Enfants Malades in Paris.

Dr. Danos also served as chief scientific officer at Genethon and senior director of research at Somatix Therapy Corporation. He has held several senior roles at the French National Centre for Scientific Research (CNRS) and at the Institut Pasteur in Paris. Dr. Danos is the former president and a founding member of the European Society of Gene and Cell Therapy.

Dr. Danos received a Masters in Genetics and Molecular Biology at University of ParisOrsay, and his Ph.D. in Biology at the Pasteur Institute and University of Paris Diderot.

Originally posted here:
BUSINESS WIRE: Olivier Danos Joins Biogen Idec as Senior Vice President, Gene Therapy

Recommendation and review posted by Bethany Smith

Stem cells and gene hold promising treatment options for Parkinson's, mandate doctors across the globe, including from Mumbai. Eleven trials to test stem cell and gene therapy for treating Parkinson's are underway currently of which the one in Mumbai had to be put on hold due to regulatory hurdles.

Currently, neuro-augmentative therapies such as usage of drugs or deep brain stimulation (DBS) are being used to treat Parkinson's disorder. "The future holds hope for neuro-restorative therapies like that of stem cells or gene infusion in the Parkinson's disorder treatment. It involves restoration of brain function to normal. In the next five to seven years, this may pave the way for future," said Dr Paresh Doshi, neurologist at Jaslok Hospital, Peddar Road in Mumbai.

Regulatory hurdles and resource constraints though have led to these trials being held up in Mumbai. Dr Doshi said that trials of Duodopa therapy which involves infusion of an active ingredient gel called Levodopa in the intestines has been kept on hold at the moment at privately-run Jaslok Hospital due to regulatory hurdles. The hospital was the only centre in entire South East Asia to have been running the trial.

"Levodopa gets converted into dopamine in the body. Normal levels of dopamine control Parkinsons disorder," said Dr Doshi.

Trials to infuse stem cells from the patient's body in the patient itself had been underway in small group of patients in India, but due to inability to recruit more patients, the trial was stopped. "We could only recruit four patients for two years. However, a similar trial is underway in China and another trial which explores adipose tissue stem cells in treating Parkinson's disease is underway in South Africa," said Dr Doshi.

In January this year, medical journal The Lancet reported that after sixteen years of trials, gene therapy is showing promising results in humans. "Three genes that promote the formation of dopamine generating cells in the brain were injected in the brain bound with a viral vector in fifteen patients. The genes are intended to boost the production of dopamine, a chemical that becomes deficient in patients withParkinson's," said The Lancet report.

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Gene therapy, stem cell therapy trials underway

Recommendation and review posted by Bethany Smith

Dr. Lam Discusses PRP, ACell, and ATP at the Hair Transplant 360 Course
http://www.hairtx.com Dallas hair transplant surgeon, Dr. Sam Lam, discusses Regenerative Medicine, including PRP, ACell, ATP at his 6th Annual Hair Transplant 360 Course at St. Louis University...

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Dr. Lam Discusses PRP, ACell, and ATP at the Hair Transplant 360 Course - Video

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Current state of personalized medicine
Brian Bolwell, MD, Chair, Taussig Cancer Institute, discusses the current state of personalized medicine. Visit Cleveland Clinic: http://bit.ly/XlxDfr Visit Health Hub from Cleveland...

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Current state of personalized medicine - Video

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C-5 C6 spinal cord injury First time walked in with a forea
This video was made October 15, 2013. This was my first time walking with a forearm crutches. Not doing too good but not bad at all either.

By: Sarah Benj

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C-5 C6 spinal cord injury First time walked in with a forea - Video

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NexImmune - Company Presentation
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San Diego, California (PRWEB) October 28, 2014

The top stem cell clinic in San Diego, Telehealth, is now offering regenerative medicine procedures for the knee to help restore cartilage and avoid the need for joint replacement. The procedures are outpatient and performed by Board Certified doctors at Telehealth. Call (888) 828-4575 for more information and scheduling.

Hundreds of thousands of knee replacements are performed every year in the US, with most being extremely successful. However, it is a major surgery and there is a chance of complications such as infection or blood clot. Therefore, it is advisable to consider a stem cell procedure for the arthritic knee in an effort to delay or avoid the procedure.

Telehealth provides the procedures with several options, including platelet rich plasma therapy, bone marrow or fat derived stem cells, along with amniotic derived procedures. All of the procedures are outpatient and low risk.

In most cases, the procedures are covered in whole or partly by insurance. Telehealth will perform an insurance verification prior to one's procedure. The Board Certified doctors at the stem cell clinic in San Diego treat patients from a broad area in Southern California. There are several locations including La Jolla, Orange and Upland CA.

In addition to stem cell procedures for knee arthritis, TeleHealth also provides regenerative medicine options for tendon and ligament injuries, sports injuries along with hip, shoulder and ankle arthritis.

For those interested in avoiding knee replacement with a procedure that can potentially preserve or repair arthritic cartilage, call Telehealth at (888) 828-4575 and visit http://stemcelltherapyincalifornia.com/ for more information.

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San Diego Stem Cell Clinic, Telehealth, Now Offering Knee Procedures for Cartilage Restoration

Recommendation and review posted by simmons